Summary

The 7 major Rett syndrome markets reached a value of US$ 182.6 Million in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 419.4 Million by 2034, exhibiting a growth rate (CAGR) of 7.85% during 2024-2034.

The Rett syndrome market has been comprehensively analyzed in IMARC's new report titled "Rett Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Rett syndrome is a rare inherited neurological and developmental illness that causes severe impairments in the brain. The disorder is associated with many mutations in the MECP2 gene, which encodes the methyl-CpG binding protein-2 (MECP2) essential for the normal function of nerve cells. These variations result in a progressive loss of language and motor skills. The symptoms associated with Rett syndrome include a wide-based gait, walking on the toes, slowed head growth, teeth grinding, difficulty chewing, diminished eye contact, apraxia, loss of muscle tone, distinctive hand movements, etc. Individuals suffering from the disorder may also experience sleep problems, seizures, cognitive disabilities, scoliosis, and breathing difficulties. The diagnosis of this disease is based on identifying underlying symptoms and evaluating the patient's physical and neurological status. A blood or genetic workup is utilized to determine the presence of gene mutations that cause Rett syndrome. Additionally, the healthcare provider may perform various procedures, such as neuroimaging and electrocardiography, to confirm a diagnosis.

The rising prevalence of inherited disorders due to gene mutations, DNA abnormalities, and chromosomal damage is primarily driving the Rett syndrome market. Apart from this, the widespread adoption of effective drugs, such as anticonvulsants and sedatives, to produce calmness, control seizures, and relieve symptoms in patients is also creating a positive outlook for the market. Additionally, the inflating application of novel diagnostic procedures, including denaturing high-performance liquid chromatography and direct sequencing analysis, which identify the gene mutations and aid in the early diagnosis of the ailment, is further propelling the market growth. Moreover, the emerging popularity of occupational and physical therapies that help improve fine motor skills, restore muscle strength, and enhance mobility in patients is acting as another significant growth-inducing factor. Besides this, the increasing utilization of nutritional interventions, such as supplements or dietary changes, to manage symptoms related to feeding difficulties or gastrointestinal issues is also bolstering the market growth. Furthermore, the escalating demand for insulin-like growth factors owing to their numerous advantages, including inhibition of neuroinflammation, decreased overactivity of microglia, correcting deficits in synaptic functions, etc., is expected to drive the Rett syndrome market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the Rett syndrome market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for Rett syndrome and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Rett syndrome market in any manner.

Time Period of the Study

Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2034

Countries Covered

United States
Germany
France
United Kingdom
Italy
Spain
Japan

Analysis Covered Across Each Country

Historical, current, and future epidemiology scenario
Historical, current, and future performance of the Rett syndrome market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the Rett syndrome market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current Rett syndrome marketed drugs and late-stage pipeline drugs.

In-Market Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:
Market Insights

How has the Rett syndrome market performed so far and how will it perform in the coming years?
What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
What was the country-wise size of the Rett syndrome market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the Rett syndrome market across the seven major markets and what will be the expected growth over the next ten years?
What are the key unmet needs in the market?

Epidemiology Insights

What is the number of prevalent cases (2018-2034) of Rett syndrome across the seven major markets?
What is the number of prevalent cases (2018-2034) of Rett syndrome by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of Rett syndrome by gender across the seven major markets?
What is the number of prevalent cases (2018-2034) of Rett syndrome by type across the seven major markets?
How many patients are diagnosed (2018-2034) with Rett syndrome across the seven major markets?
What is the size of the Rett syndrome patient pool (2018-2023) across the seven major markets?
What would be the forecasted patient pool (2024-2034) across the seven major markets?
What are the key factors driving the epidemiological trend of Rett syndrome?
What will be the growth rate of patients across the seven major markets?

Rett Syndrome: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

What are the current marketed drugs and what are their market performance?
What are the key pipeline drugs and how are they expected to perform in the coming years?
How safe are the current marketed drugs and what are their efficacies?
How safe are the late-stage pipeline drugs and what are their efficacies?
What are the current treatment guidelines for Rett syndrome drugs across the seven major markets?
Who are the key companies in the market and what are their market shares?
What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Rett syndrome market?
What are the key regulatory events related to the Rett syndrome market?
What is the structure of clinical trial landscape by status related to the Rett syndrome market?
What is the structure of clinical trial landscape by phase related to the Rett syndrome market?
What is the structure of clinical trial landscape by route of administration related to the Rett syndrome market?

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Table of Contents

1 Preface

2 Scope and Methodology
2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
2.3.1 Primary Sources
2.3.2 Secondary Sources
2.4 Market Estimation
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Forecasting Methodology

3 Executive Summary

4 Rett Syndrome - Introduction
4.1 Overview
4.2 Regulatory Process
4.3 Epidemiology (2018-2023) and Forecast (2024-2034)
4.4 Market Overview (2018-2023) and Forecast (2024-2034)
4.5 Competitive Intelligence

5 Rett Syndrome - Disease Overview
5.1 Introduction
5.2 Symptoms and Diagnosis
5.3 Pathophysiology
5.4 Causes and Risk Factors
5.5 Treatment

6 Patient Journey

7 Rett Syndrome - Epidemiology and Patient Population
7.1 Epidemiology - Key Insights
7.2 Epidemiology Scenario - Top 7 Markets
7.2.1 Epidemiology Scenario (2018-2023)
7.2.2 Epidemiology Forecast (2024-2034)
7.2.3 Epidemiology by Age (​2018-2034​)
7.2.4 Epidemiology by Gender (​2018-2034​)
7.2.5 Epidemiology by Type (​2018-2034​)
7.2.6 Diagnosed Cases (​2018-2034​)
7.2.7 Patient Pool/Treated Cases (​2018-2034​)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023)
7.3.2 Epidemiology Forecast (2024-2034)
7.3.3 Epidemiology by Age (​2018-2034​)
7.3.4 Epidemiology by Gender (​2018-2034​)
7.3.5 Epidemiology by Type (​2018-2034​)
7.3.6 Diagnosed Cases (​2018-2034​)
7.3.7 Patient Pool/Treated Cases (​2018-2034​)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario (2018-2023)
7.4.2 Epidemiology Forecast (2024-2034)
7.4.3 Epidemiology by Age (​2018-2034​)
7.4.4 Epidemiology by Gender (​2018-2034​)
7.4.5 Epidemiology by Type (​2018-2034​)
7.4.6 Diagnosed Cases (​2018-2034​)
7.4.7 Patient Pool/Treated Cases (​2018-2034​)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023)
7.5.2 Epidemiology Forecast (2024-2034)
7.5.3 Epidemiology by Age (​2018-2034​)
7.5.4 Epidemiology by Gender (​2018-2034​)
7.5.5 Epidemiology by Type (​2018-2034​)
7.5.6 Diagnosed Cases (​2018-2034​)
7.5.7 Patient Pool/Treated Cases (​2018-2034​)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023)
7.6.2 Epidemiology Forecast (2024-2034)
7.6.3 Epidemiology by Age (​2018-2034​)
7.6.4 Epidemiology by Gender (​2018-2034​)
7.6.5 Epidemiology by Type (​2018-2034​)
7.6.6 Diagnosed Cases (​2018-2034​)
7.6.7 Patient Pool/Treated Cases (​2018-2034​)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023)
7.7.2 Epidemiology Forecast (2024-2034)
7.7.3 Epidemiology by Age (​2018-2034​)
7.7.4 Epidemiology by Gender (​2018-2034​)
7.7.5 Epidemiology by Type (​2018-2034​)
7.7.6 Diagnosed Cases (​2018-2034​)
7.7.7 Patient Pool/Treated Cases (​2018-2034​)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023)
7.8.2 Epidemiology Forecast (2024-2034)
7.8.3 Epidemiology by Age (​2018-2034​)
7.8.4 Epidemiology by Gender (​2018-2034​)
7.8.5 Epidemiology by Type (​2018-2034​)
7.8.6 Diagnosed Cases (​2018-2034​)
7.8.7 Patient Pool/Treated Cases (​2018-2034​)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023)
7.9.2 Epidemiology Forecast (2024-2034)
7.9.3 Epidemiology by Age (​2018-2034​)
7.9.4 Epidemiology by Gender (​2018-2034​)
7.9.5 Epidemiology by Type (​2018-2034​)
7.9.6 Diagnosed Cases (​2018-2034​)
7.9.7 Patient Pool/Treated Cases (​2018-2034​)

8 Rett Syndrome - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Rett Syndrome - Unmet Needs

10 Rett Syndrome - Key Endpoints of Treatment

11 Rett Syndrome - Marketed Products
11.1 List of Rett Syndrome Marketed Drugs Across the Top 7 Markets
11.1.1 Daybue (Trofinetide) - ACADIA Pharmaceuticals/Neuren Pharmaceuticals
11.1.1.1 Drug Overview
11.1.1.2 Mechanism of Action
11.1.1.3 Regulatory Status
11.1.1.4 Clinical Trial Results
11.1.1.5 Sales Across Major Markets

Kindly note that the above only represents a partial list of marketed drugs, and the complete list has been provided in the report.

12 Rett Syndrome - Pipeline Drugs
12.1 List of Rett Syndrome Pipeline Drugs Across the Top 7 Markets
12.1.1 ANAVEX2-73 - Anavex Life Sciences
12.1.1.1 Drug Overview
12.1.1.2 Mechanism of Action
12.1.1.3 Clinical Trial Results
12.1.1.4 Safety and Efficacy
12.1.1.5 Regulatory Status
12.1.2 TSHA-102 - Taysha Gene Therapies
12.1.2.1 Drug Overview
12.1.2.2 Mechanism of Action
12.1.2.3 Clinical Trial Results
12.1.2.4 Safety and Efficacy
12.1.2.5 Regulatory Status
12.1.3 VYNT-0126- Vyant Bio
12.1.3.1 Drug Overview
12.1.3.2 Mechanism of Action
12.1.3.3 Clinical Trial Results
12.1.3.4 Safety and Efficacy
12.1.3.5 Regulatory Status
12.1.4 NNZ-2566- Neuren Pharmaceuticals Limited
12.1.4.1 Drug Overview
12.1.4.2 Mechanism of Action
12.1.4.3 Clinical Trial Results
12.1.4.4 Safety and Efficacy
12.1.4.5 Regulatory Status

Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report.

13. Rett Syndrome - Attribute Analysis of Key Marketed and Pipeline Drugs

14. Rett Syndrome – Clinical Trial Landscape
14.1 Drugs by Status
14.2 Drugs by Phase
14.3 Drugs by Route of Administration
14.4 Key Regulatory Events

15 Rett Syndrome - Market Scenario
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Rett Syndrome - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Rett Syndrome - Market Size by Therapies
15.2.2.1 Market Size by Therapies (2018-2023)
15.2.2.2 Market Forecast by Therapies (2024-2034)
15.3 Market Scenario - United States
15.3.1 Rett Syndrome - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Rett Syndrome - Market Size by Therapies
15.3.2.1 Market Size by Therapies (2018-2023)
15.3.2.2 Market Forecast by Therapies (2024-2034)
15.3.3 Rett Syndrome - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Rett Syndrome - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Rett Syndrome - Market Size by Therapies
15.4.2.1 Market Size by Therapies (2018-2023)
15.4.2.2 Market Forecast by Therapies (2024-2034)
15.4.3 Rett Syndrome - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Rett Syndrome - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Rett Syndrome - Market Size by Therapies
15.5.2.1 Market Size by Therapies (2018-2023)
15.5.2.2 Market Forecast by Therapies (2024-2034)
15.5.3 Rett Syndrome - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Rett Syndrome - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Rett Syndrome - Market Size by Therapies
15.6.2.1 Market Size by Therapies (2018-2023)
15.6.2.2 Market Forecast by Therapies (2024-2034)
15.6.3 Rett Syndrome - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Rett Syndrome - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Rett Syndrome - Market Size by Therapies
15.7.2.1 Market Size by Therapies (2018-2023)
15.7.2.2 Market Forecast by Therapies (2024-2034)
15.7.3 Rett Syndrome - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Rett Syndrome - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Rett Syndrome - Market Size by Therapies
15.8.2.1 Market Size by Therapies (2018-2023)
15.8.2.2 Market Forecast by Therapies (2024-2034)
15.8.3 Rett Syndrome - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Rett Syndrome - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Rett Syndrome - Market Size by Therapies
15.9.2.1 Market Size by Therapies (2018-2023)
15.9.2.2 Market Forecast by Therapies (2024-2034)
15.9.3 Rett Syndrome - Access and Reimbursement Overview

16 Rett Syndrome - Recent Events and Inputs From Key Opinion Leaders

17 Rett Syndrome Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Rett Syndrome Market – Strategic Recommendations

19 Appendix